Bay Writing, Copenhagen, Denmark.
Pfizer Denmark, Ballerup, Denmark.
ESC Heart Fail. 2022 Jun;9(3):1524-1541. doi: 10.1002/ehf2.13884. Epub 2022 Mar 27.
Wild-type transthyretin amyloid cardiomyopathy (ATTRwt CM) is a more common disease than previously thought. Awareness of ATTRwt CM and its diagnosis has been challenged by its unspecific and widely distributed clinical manifestations and traditionally invasive diagnostic tools. Recent advances in echocardiography and cardiac magnetic resonance (CMR), non-invasive diagnosis by bone scintigraphy, and the development of disease-modifying treatments have resulted in an increased interest, reflected in multiple publications especially during the last decade. To get an overview of the scientific knowledge and gaps related to patient entry, suspicion, diagnosis, and systematic screening of ATTRwt CM, we developed a framework to systematically map the available evidence of (i) when to suspect ATTRwt CM in a patient, (ii) how to diagnose the disease, and (iii) which at-risk populations to screen for ATTRwt CM. Articles published between 2010 and August 2021 containing part of or a full diagnostic pathway for ATTRwt CM were included. From these articles, data for patient entry, suspicion, diagnosis, and screening were extracted, as were key study design and results from the original studies referred to. A total of 50 articles met the inclusion criteria. Of these, five were position statements from academic societies, while one was a clinical guideline. Three articles discussed the importance of primary care providers in terms of patient entry, while the remaining articles had the cardiovascular setting as point of departure. The most frequently mentioned suspicion criteria were ventricular wall thickening (44/50), carpal tunnel syndrome (42/50), and late gadolinium enhancement on CMR (43/50). Diagnostic pathways varied slightly, but most included bone scintigraphy, exclusion of light-chain amyloidosis, and the possibility of doing a biopsy. Systematic screening was mentioned in 16 articles, 10 of which suggested specific at-risk populations for screening. The European Society of Cardiology recommends to screen patients with a wall thickness ≥12 mm and heart failure, aortic stenosis, or red flag symptoms, especially if they are >65 years. The underlying evidence was generally good for diagnosis, while significant gaps were identified for the relevance and mutual ranking of the different suspicion criteria and for systematic screening. Conclusively, patient entry was neglected in the reviewed literature. While multiple red flags were described, high-quality prospective studies designed to evaluate their suitability as suspicion criteria were lacking. An upcoming task lies in defining and evaluating at-risk populations for screening. All are steps needed to promote early detection and diagnosis of ATTRwt CM, a prerequisite for timely treatment.
野生型转甲状腺素蛋白淀粉样心肌病(ATTRwt CM)比以前认为的更为常见。由于其临床表现不具特异性且分布广泛,传统的有创诊断工具也存在局限性,因此人们对 ATTRwt CM 的认识和诊断一直存在挑战。近年来,超声心动图和心脏磁共振(CMR)的进展、骨闪烁扫描的非侵入性诊断以及疾病修饰治疗的发展,增加了人们对该病的兴趣,这在过去十年中的多项出版物中都有所体现。为了全面了解与 ATTRwt CM 患者纳入、怀疑、诊断和系统筛查相关的科学知识和差距,我们制定了一个框架,以系统地绘制(i)何时怀疑患者患有 ATTRwt CM,(ii)如何诊断该疾病,以及(iii)筛查哪些高危人群患有 ATTRwt CM 的可用证据。纳入了 2010 年至 2021 年 8 月期间发表的部分或完整的 ATTRwt CM 诊断途径的文章。从这些文章中提取了与患者纳入、怀疑、诊断和筛查相关的数据,以及原始研究中提到的关键研究设计和结果。共有 50 篇文章符合纳入标准。其中,有 5 篇是学术协会的立场声明,还有 1 篇是临床指南。有 3 篇文章讨论了初级保健提供者在患者纳入方面的重要性,而其余文章则以心血管环境为起点。最常提到的怀疑标准是心室壁增厚(44/50)、腕管综合征(42/50)和 CMR 上的晚期钆增强(43/50)。诊断途径略有不同,但大多数包括骨闪烁扫描、排除轻链淀粉样变性和进行活检的可能性。16 篇文章中提到了系统筛查,其中 10 篇文章建议对特定的高危人群进行筛查。欧洲心脏病学会建议对壁厚度≥12mm 和心力衰竭、主动脉瓣狭窄或有红色标志症状的患者进行筛查,尤其是年龄>65 岁的患者。诊断的基础证据总体较好,但不同怀疑标准的相关性和相互排序以及系统筛查方面仍存在显著差距。总之,文献中忽视了患者纳入。尽管描述了多个红色标志,但缺乏设计用于评估其作为怀疑标准的适用性的高质量前瞻性研究。即将面临的任务是定义和评估筛查的高危人群。所有这些步骤都有助于促进 ATTRwt CM 的早期发现和诊断,这是及时治疗的前提。